Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Long-Term Follow-up of AAV8-Mediated G6Pase Gene Transfer in Adults with GSDIa
2023-504004-29-00Phase 2Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Feb 2024
- Modality
- Gene Therapies
- Countries
- 4
01
Overview
Phase 2, open-label, single-arm trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Incidence of adverse events and serious adverse events. Started Feb 2024; status: completed.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
Beyond the preview
Go deeper on 2023-504004-29-00.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2023-504004-29-00.